Table 2 Selected studies on the novel targeted therapeutic in combination with RUX on myelofibrosis from the 2022 ASH annual meeting
Name | Target | Route | Trial Phase | Inclusion Criteria (prior treatments; subject age; spleen size) | Subject Baseline Characteristics | Study Duration (current /planned) | Accrual (current accrual #/target accrual #) | Efficacy | Adverse Events | Clinicaltrials.gov Registration | References |
|---|---|---|---|---|---|---|---|---|---|---|---|
SIR | HDM2 | PO | Ib | One prior JAKi; ≥ 18 years; palpable spleen ≥ 5 cm or SV ≥ 450 cm3 | Median spleen volume of 1162 cm3 | 30/45 months | 23/45 | SVR35 (100%) at W24 | Fatigue GI toxicity Anemia Leukopenia Thrombocytopenia | NCT04097821 | [8] |
PELA | BET | PO | II | One prior JAKi; ≥ 18 years; SV ≥ 450 cm3 | Median age of 68 years | 96/123 months | 84/341 | SVR35 (80%); MF-SAF-TSS50 (81%); BMF (40% ≥ 1 grade improvement) at any time | Thrombocytopenia Anemia | NCT02158858 | [9] |
Navitoclax | BCL-2 family | PO | II | One Prior IAKi or JAKi-naïve patients; ≥ 18 years; splenomegaly | JAKi-naïve pts; median age of 69 years; median SV of 1889 cm3 | 51/131 months | 32/191 | SVR35(59%) in high DIPSS score pts, BMF (35%) at W24 | Not reported | NCT03222609 | [10] |
Parsaclisib | PI3K | PO | II | Pts with existing stable regimen of RUX; ≥ 18 years; palpable spleen ≥ 10 cm | Median age of 68 years; 47% of pts male; median SV of 2415 cm3 in QD/QW, 1878 cm3 in QD | 63/63 months | 74/74 | SVR35 (7.1%); MPN-SAF-TSS50 (48.6%); BMF (33%) all daily dosing at W24 | Pneumonia Fatigue Hypoxia Dyspnea Elevation of liver enzymes Hypocalcemia Thrombocytopenia | NCT02718300 | [11] |
SEL | XPO1 | PO | I/II | JAKi-naïve pts; ≥ 18 years; SV ≥ 450 cm3 | Median age of 64 years | 17/44 months | 19/237 | SVR35 (84%) at any time; TSS50 (69%) at W12 | Nausea Anemia Vomiting Thrombocytopenia | NCT04562389 | [12] |